Tofacitinib Approved for Polyarticular Juvenile Idiopathic Arthritis


The FDA has approved tofacitinib for the treatment of children and adolescents 2 years and older with active polyarticular course juvenile idiopathic arthritis.

The FDA has approved tofacitinib (Xeljanz, Pfizer) for the treatment of children and adolescents 2 years of age and older with active polyarticular course juvenile idiopathic arthritis (pcJIA). Tofacitinib was approved in both a tablet and oral solution, which are administered based on weight.

pcJIA causes significant joint pain, which can limit participation in child appropriate activates. Although several treatment options already exist, tofacitinib does not require an injection, which can be burdensome to both patients and their caregivers.

pcJIA begins before 16 years of age and is characterized as arthritis in 5 or more joints, affecting both small and large joints. Approximately 300,000 children in the United States are living with a form of juvenile idiopathic arthritis.

Tofacitinib’s approval is based on a phase 3 open-label study comprised of 2 phases. The first was an 18-week, open-label, run-in phase that included 225 patients. The second lasted for 26 weeks and was a double-blind, placebo-controlled, randomized, withdrawal phase that included 173 patients. Patients received either a 5 mg tablet or a 1 mg/ml oral solution twice daily was based on their weight, as those who received the oral solution weighed less than 40 kg.

Of the patients treated with tofacitinib, 31% had a disease flare during the 44-week trial period, whereas 55% of the patients treated with the placebo had a disease flare, according to the study. The study defined flare as a 30% or more worsening in at least 3 of the 6 variables of the JIA ACR core set with only 1 of the remaining 3 variables improving.

“Many children and adolescents living with polyarticular course juvenile idiopathic arthritis, or pcJIA, are in need of advanced oral treatment options, so we are proud to now offer Xeljanz to this patient community,” Michael Corbo, chief development officer of Inflammation and Immunology, Pfizer Global Product Development, said in the press release. “This approval, which is the fourth indication for XELJANZ, reinforces its utility in the treatment of immune-mediated inflammatory conditions and further demonstrates our expertise in JAK science.”

Patients taking tofacitinib are at an increased risk of serious infections, such as active tuberculosis, invasive fungal infections, and herpes zoster. If a serious infection develops, tofacitinib should be interrupted until the infection is controlled.


U.S. FDA Approves Pfizer’s XELJANZ® (tofacitinib) for the Treatment of Active Polyarticular Course Juvenile Idiopathic Arthritis [News Release] September 28, 2020; New York, NY. Accessed September 29, 2020.

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